Neu­rotrope bat­tered in lat­est Alzheimer's flop; Platelet Bio­Gen­e­sis rais­es $26M-plus

Neu­rotrope be­came the lat­est ca­su­al­ty in the high-risk Alzheimer’s R&D field. The biotech re­port­ed that their Phase II study of Bryo­statin-1 failed to show any im­prove­ment over place­bo us­ing the Se­vere Im­pair­ment Bat­tery (SIB) to­tal score at week 13. In fact, the drug arm did a lit­tle worse than a sug­ar pill, with an av­er­age in­crease of 1.3 points for the drug and 2.1 points for place­bo. Their shares $NTRP cratered in the rout that fol­lowed, plung­ing 78%. It’s cur­rent­ly a pen­ny stock.

The Alzheimer’s R&D field hasn’t record­ed a suc­cess in chang­ing the tra­jec­to­ry of the dis­ease, with re­cent piv­otal fail­ures forc­ing all the play­ers to re­con­sid­er their strate­gies.

Platelet Bio­Gen­e­sis (PBG) — a com­pa­ny fo­cused on stem cell-de­rived, on-de­mand hu­man platelets and platelet-based ther­a­peu­tics — has raised more than $26 mil­lion in Se­ries A-1 fi­nanc­ing. In com­bi­na­tion to this round, as well as Se­ries A fi­nanc­ing and grants, the com­pa­ny’s to­tal fund­ing is brought to about $45 mil­lion. The fi­nanc­ing was co-led by Ziff Cap­i­tal Part­ners and Qim­ing Ven­ture Part­ners USA in ad­di­tion to ex­ist­ing in­ves­tures Nest.Bio Ven­tures and eCoast An­gels.

The com­pa­ny said that it “will use the funds from the Se­ries A-1 round to ac­cel­er­ate the de­vel­op­ment of its donor-in­de­pen­dent platelet man­u­fac­tur­ing ca­pa­bil­i­ties and ad­vance its proof of con­cept work around platelet-based ther­a­peu­tics.” PBG plans to dou­ble its staff to ap­prox­i­mate­ly 50 em­ploy­ees in 2020.

Stu­art Levy, co-founder of Paratek Phar­ma­ceu­ti­cals, passed away last week.

“Stu­art was a dis­tin­guished physi­cian who was the fore­front of an­tibi­ot­ic de­vel­op­ment and a ded­i­cat­ed cham­pi­on for the pru­dent use of an­tibi­otics,” said Evan Loh, Paratek CEO. “More im­por­tant­ly, he was a dear friend and men­tor to so many and his pas­sion lives on in each of us as we work to con­tin­ue his mis­sion of com­bat­ing the dai­ly threat of life-threat­en­ing in­fec­tions. We will great­ly miss him.”

Levy co-found­ed Paratek more than 20 years ago along with Wal­ter Gilbert. He was in­stru­men­tal in the de­vel­op­ment of the com­pa­ny’s lead an­tibi­otics, Nuzyra and Seysara. Levy dis­cov­ered the ef­flux mech­a­nism for drug (tetra­cy­cline) re­sis­tance and was among the first sci­en­tists to doc­u­ment the trans­fer of re­sis­tant bac­te­ria from an­i­mals to farm­work­ers. His 1992 book, The An­tibi­ot­ic Para­dox: How Mir­a­cle Drugs Are De­stroy­ing the Mir­a­cle, has been cit­ed wide­ly and trans­lat­ed in­to four lan­guages.

The news comes a few weeks af­ter the com­pa­ny pub­lished pos­i­tive Phase III da­ta for Nuzyra for pa­tients with AB­SS­SI.

→ In the midst of a sur­prise in­ter partes re­view by the US Patent Tri­als Ap­peal Board (PT­AB) for Alex­ion’s Soliris, the com­pa­ny and Bridge­Bio’s sub­sidiary Ei­dos Ther­a­peu­tics have inked an agree­ment grant­i­ng Alex­ion an ex­clu­sive li­cense to de­vel­op and com­mer­cial­ize their oral­ly-ad­min­is­tered small mol­e­cule AG10 — de­signed to treat transthyretin amy­loi­do­sis (AT­TR) by bind­ing and sta­bi­liz­ing transthyretin (TTR) in the blood— in Japan.

“Ei­dos is cur­rent­ly eval­u­at­ing AG10 in a Phase III study in the US and Eu­rope for AT­TR car­diomy­opa­thy (AT­TR-CM) – a pro­gres­sive, fa­tal dis­ease caused by the ac­cu­mu­la­tion of mis­fold­ed TTR amy­loid in the heart – and plans to be­gin a Phase III study in AT­TR polyneu­ropa­thy (AT­TR-PN) – a pro­gres­sive, fa­tal dis­ease caused by the ac­cu­mu­la­tion of mis­fold­ed TTR amy­loid in the pe­riph­er­al ner­vous sys­tem.”

→ A few months af­ter Ac­celeron and its part­ner at Cel­gene se­cured pri­or­i­ty re­view for their red blood cell boost­ing drug, lus­pa­ter­cept — to man­age ane­mia re­sult­ing from myelodys­plas­tic syn­drome (MDS) as well as be­ta tha­lassemia — the com­pa­ny has an­nounced an­oth­er win. This time they’ve gained an or­phan drug des­ig­na­tion by the FDA for its in­ves­ti­ga­tion­al ther­a­py so­tater­cept for the treat­ment of pa­tients with pul­monary ar­te­r­i­al hy­per­ten­sion (PAH).

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Kristen Hege, Bristol Myers Squibb SVP, early clinical development, oncology/hematology and cell therapy (Illustration: Assistant Editor Kathy Wong for Endpoints News)

Q&A: Bris­tol My­er­s' Kris­ten Hege on cell ther­a­py, can­cer pa­tients and men­tor­ing the next gen­er­a­tion

Kristen Hege leads Bristol Myers Squibb’s early oncology discovery program carrying on from the same work at Celgene, which was acquired by BMS in 2019. She’s known for her early work in CAR-T, having pioneered the first CAR-T cell trial for solid tumors more than 25 years ago.

However, the eminent physician-scientist is more than just a drug developer mastermind. She’s also a practicing physician, mother to two young women, an avid backpacker and intersecting all those interests — a champion of young women and people of color in STEM and life sciences.

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Eisai and METAvivor plan to debut the latest 'This is MBC' campaign at the San Antonio Breast Cancer Symposium (SABCS).

Ei­sai re-ups metasta­t­ic breast can­cer aware­ness cam­paign with strik­ing pa­tient pho­tographs

Eisai is debuting the newest ads in its long-running “This is MBC” campaign this week. In what’s become an annual tradition, Eisai and metastatic breast cancer advocacy partner METAvivor will show the striking photographs of people living with metastatic breast cancer first at the San Antonio Breast Cancer Symposium (SABCS).

The new “Imagine” campaign features 12 patients photographed around waterfalls to symbolize that same kind of sudden drop into a pool that MBC causes in a person’s life, said Beth Fairchild, co-founder of #CancerCulture who was the president of METAvivor six years ago when the campaign began. Fairchild, who is living with MBC, has helped create all of the annual “This is MBC” campaigns.

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Gossamer Bio CEO Faheem Hasnain at Endpoints' #BIO22 panel (J.T. MacMillan Photography for Endpoints News)

Gos­samer’s Fa­heem Has­nain de­fends a round of pos­i­tive PAH da­ta as a clear win. But can these PhII re­sults stand up to scruti­ny?

Gossamer Bio $GOSS posted a statistically significant improvement for its primary endpoint in the key Phase II TORREY trial for lead drug seralutinib on Tuesday morning. But CEO Faheem Hasnain has some explaining to do on the important secondary of the crucial six-minute walk distance test — which will be the primary endpoint in Phase III — as the data on both endpoints fell short of expectations, missing one analyst’s bar on even modest success.

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Pfiz­er and BioN­Tech look to toss Mod­er­na patent suit, call­ing claims 'unen­force­able'

Pfizer and BioNTech took a swing at Moderna’s Covid-19 patent claims in Massachusetts federal court on Monday, calling them “invalid,” “overbroad” and “unenforceable.”

The defendants also filed counterclaims against the Cambridge, MA-based biotech, seeking a dismissal of the case, recovery of court fees and an official judgment invalidating Moderna’s claims.

Moderna sued Pfizer and BioNTech back in August, alleging that the partners’ Covid-19 vaccine Comirnaty copied parts of Moderna’s vaccine technology patented before the pandemic, when it was developing an mRNA vaccine for MERS, another respiratory illness.

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Rick Modi, Affinia Therapeutics CEO

Ver­tex-part­nered gene ther­a­py biotech Affinia scraps IPO plans

Affinia Therapeutics has ditched its plans to go public in a relatively closed-door market that has not favored Nasdaq debuts for the drug development industry most of this year. A pandemic surge in 2020 and 2021 opened the doors for many preclinical startups, which caught Affinia’s attention and gave the gene therapy biotech confidence in the beginning days of 2022 to send in its S-1.

But on Friday, Affinia threw in the S-1 towel and concluded now is not the time to step onto Wall Street. The biotech has put out few public announcements since the spring of this year. Endpoints News picked the startup as one of its 11 biotechs to watch last year.

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Glen­mark hit with warn­ing let­ter over pro­ce­dures, qual­i­ty con­trol is­sues at In­dia man­u­fac­tur­ing plant

The generics producer Glenmark Pharmaceuticals has been handed a warning letter by US regulators.

The letter, which was sent to the manufacturer on Nov. 22, noted issues from an inspection over the summer at Glenmark’s facility in the town of Colvale, India, in the state of Goa.

According to the letter, the FDA found that Glenmark’s investigation of rejected batches of drugs “failed to extend to other batches, dosage strengths, and drug products.” The warning letter also noted that the site had failed to establish “adequate written procedures” for production and process control to ensure drugs have the correct strength, quality and purity.

Klick Health is lighting the way, literally, this holiday season to encourage connection for lonely seniors in long-term care facilities.

Klick Health an­nu­al hol­i­day spot­light se­nior lone­li­ness and the pow­er of con­nec­tion

Every year Klick Health leans into a cause for the holidays, and this year it’s highlighting the sometimes lonely season for seniors. So Klicksters, as employees call themselves, decided to brighten one nursing home community in hopes of inspiring others to do the same.

Klick literally lit up the Tony Stacey Centre for Veterans Care, a long-term care home in Toronto where 75% of residents receive no visitors during the holiday season. The agency brought staff and family along with lighting crews and musicians for a “Light the Way” event, creating a video of the experience debuting on Tuesday.

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Sum­i­to­vant sub­sidiaries En­zy­vant and Al­ta­vant merge in­to com­bined com­pa­ny

Two Sumitovant Biopharma entities are merging under one name, effective immediately.

Enzyvant Therapeutics and Altavant Sciences announced they have merged to form a singular entity focused on developing therapies for patients with rare diseases. The combined company will keep the name Enzyvant and along with clinical development will eventually include in-house manufacturing.

Bill Symonds, the current CEO of both Altavant and Enzyvant, is now CEO of the merged company.