Strug­gling Heat gets a warm re­sponse to a snap­shot on can­cer vax da­ta

Jeff Hutchins, Heat Bi­o­log­ics

Heat Bi­o­log­ics man­aged to at least tem­porar­i­ly ex­tri­cate it­self from pen­ny stock ter­ri­to­ry af­ter of­fer­ing a pos­i­tive up­date on its com­bi­na­tion study for non-small cell lung can­cer. Its stock surged more than 20% this morn­ing based on an up­date for a hand­ful of pa­tients tak­ing a com­bi­na­tion of HS-110 and Bris­tol-My­ers Squibb’s check­point in­hibitor Op­di­vo.

The up­dat­ed snap­shot from the Durham, NC-based biotech in­clud­ed a note that 3 of 5 pa­tients en­rolled in a co­hort for low tu­mor in­fil­trat­ing lym­pho­cytes “ex­pe­ri­enced sig­nif­i­cant tu­mor re­duc­tion, which is high­er than the 10% re­sponse rate of low TIL pa­tients re­port­ed for ex­ist­ing da­ta on nivolum­ab alone.” 5 of 12 evalu­able pa­tients ex­pe­ri­enced an im­mune re­sponse to the com­bo.

As a re­sult Heat’s stock $HBTX nosed above the $1 mark.

Heat has ex­pe­ri­enced re­peat­ed set­backs that ground down its share price.  Last spring, the biotech cut staff. Then in the fall their drug HS-410 failed a mid-stage study for blad­der can­cer. The com­pa­ny cur­rent­ly has a mi­cro mar­ket cap hov­er­ing around $24 mil­lion, putting it in a very vul­ner­a­ble group. And Nas­daq is­sued a delist­ing warn­ing just a few days ago.

“Check­point in­hibitors, such as nivolum­ab, are cur­rent­ly ef­fec­tive in treat­ing ap­prox­i­mate­ly 10% of lung can­cer pa­tients with low TIL and about 20% of pa­tients over­all in the 2nd line set­ting,” said Jeff Hutchins, Heat’s top sci­en­tist and SVP, Pre­clin­i­cal De­vel­op­ment. “Our re­sults ap­pear to fur­ther val­i­date the ex­pect­ed mech­a­nism of ac­tion of our ap­proach in com­bi­na­tion with check­point in­hibitors, with a con­tin­u­ing trend to­wards ear­ly and sus­tained T cell ac­ti­va­tion in the pe­riph­er­al blood cells.”

Inside FDA HQ (File photo)

The FDA just ap­proved the third Duchenne MD drug. And reg­u­la­tors still don’t know if any of them work

Last year Sarepta hit center stage with the FDA’s controversial reversal of its CRL for the company’s second Duchenne muscular dystrophy drug — after the biotech was ambushed by agency insiders ready to reject a second pitch based on the same disease biomarker used for the first approval for eteplirsen, without actual data on the efficacy of the drug.

On Wednesday the FDA approved the third Duchenne MD drug, based on the same biomarker. And regulators were ready to act yet again despite the lack of efficacy data.

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Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Franz-Werner Haas, CureVac CEO

UP­DAT­ED: On the heels of a snap $1B raise, Cure­Vac out­lines plans to seek emer­gency OK for Covid-19 vac­cine -- shares rock­et up

CureVac is going from being one of the quietest players in the race to develop a new vaccine to fight the worst public health crisis in a century to a challenger for the multibillion-dollar market that awaits the first vaccines to make it over the finish line. Typically low-key at a time of brash comments and incredibly ambitious development timelines from the leaders, CureVac now is jumping straight into the spotlight.

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US gov­ern­ment re­port­ed­ly be­gins prepar­ing for Covid-19 chal­lenge tri­als. Are they eth­i­cal?

Controversial human challenge trials for potential Covid-19 vaccines reportedly have a new booster — the US government.

Scientists working for the government have begun manufacturing a strain of the novel coronavirus that could be used in such studies, Reuters reported Friday morning. The trials would enroll healthy volunteers to be vaccinated and then intentionally infected with a weakened coronavirus.

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Trevor Martin (Mammoth)

Eye­ing in-vi­vo edit­ing, Mam­moth li­cens­es Jen­nifer Doud­na’s new CRISPR en­zyme

Last month, Jennifer Doudna revealed in Science a new, “hyper-compact” CRISPR enzyme that was half the size of traditional CRISPR enzymes and could, she suspected, offer a new, more versatile tool for gene editing.

Now, the University of California-Berkeley has licensed that enzyme, known as Casφ, exclusively to a biotech startup she and two former students set up three years ago: Mammoth Biosciences. It’s the second new CRISPR protein Mammoth has licensed from Doudna’s lab, after they licensed Cas14 in 2019.

Sanofi vet Kather­ine Bowdish named CEO of PIC Ther­a­peu­tics; As the world Terns: Liv­er dis­ease biotech makes ex­ec­u­tive changes

PIC Therapeutics hasn’t raised much money, yet. But the fledgling biotech has attracted a high-profile player to the helm.

The Boston-based biotech has handed the reins to Katherine Bowdish as its president and CEO. Bowdish will also join the board of directors of PIC. Bowdish joins from Sanofi where she served as VP and head of R&D strategy, as well as helping launch and lead Sanofi Sunrise, a venture investment and partnering vehicle at Sanofi. Before that, Bowdish held several exec roles at Permeon Biologics, Anaphore, Alexion Pharmaceuticals and Prolifaron (acquired by Alexion).

Cal­lid­i­tas bets up to $102M on a biotech buy­out, snag­ging a once-failed PBC drug

After spending years developing its oral formulation of the corticosteroid budesonide, Sweden’s Calliditas now has its sights set on the primary biliary cholangitis field.

The company will buy out France-based Genkyotex, and it’s willing to bet up to €87 million ($102 million) that Genkyotex’s failed Phase II drug, GKT831, will do better in late-stage trials.

Under the current agreement, Calliditas $CALT will initially pay €20.3 million in cash for 62.7% of Genkyotex (or €2.80 a piece for 7,236,515 shares) in early October, then circle back for the rest of Genkyotex’s shares under the same terms. If nothing changes, the whole buyout will cost Calliditas €32.3 million, plus up to  €55 million in contingent rights.

James Wilson, WuXi Global Forum at JPM20

FDA puts up a red light for Pas­sage Bio’s first gene ther­a­py pro­gram, de­lay­ing a pro­gram from James Wilson's group at Penn

Gene therapy pioneer James Wilson spearheaded animal studies demonstrating the potential of new treatments injected directly into the brain, looking to jumpstart a once-and-done fix for an extraordinarily rare disease called GM1 gangliosidosis in infants. His team at the University of Pennsylvania published their work on monkeys and handed it over to Passage Bio, a Wilson-inspired startup building a pipeline of gene therapies — with an IND for PBGM01 to lead the way.

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Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.