Heron's pain drug nails PhI­II, prep­ping the com­pa­ny for NDA lat­er this year

San Diego’s Heron Ther­a­peu­tics $HRTX is on a roll, to­day trum­pet­ing good news from its Phase III tri­al in post-op­er­a­tive pain. The promis­ing da­ta will back the drug­mak­er’s bid for FDA ap­proval of the drug lat­er this year. If ap­proved, it would be the sec­ond FDA OK for Heron in a mat­ter of months.

The news is send­ing Heron’s stock price soar­ing, up near­ly 26% since the an­nounce­ment.

The com­pa­ny’s pain drug is a long-act­ing lo­cal anes­thet­ic made up of bupi­va­caine and meloxi­cam, which Heron calls HTX-011. The drug was test­ed in two Phase III stud­ies that mea­sured post-surgery pain and opi­oid use af­ter bunionec­to­my (bunion re­moval) and her­nia re­pair. HTX-011 achieved all its pri­ma­ry and sec­ondary end­points in both tri­als, ac­cord­ing to Heron.

Bar­ry Quart

“If ap­proved, we be­lieve that HTX-011 could have a sig­nif­i­cant im­pact on the opi­oid cri­sis by re­duc­ing the use of opi­oids af­ter surgery, while at the same time al­low­ing pa­tients to ex­pe­ri­ence less pain,” said Heron’s CEO Bar­ry Quart in a state­ment.

In the bunion tri­al, pa­tients tak­ing HTX-011 saw a 27% drop in pain in­ten­si­ty com­pared to the place­bo group, and an 18% drop com­pared to the group tak­ing bupi­va­caine, the cur­rent stan­dard-of-care. Pa­tients on the drug al­so used 37% few­er opi­oids than place­bo pa­tients and 25% few­er than bupi­va­caine pa­tients.

In the her­nia tri­al, pa­tients on Heron’s drug saw a 23% re­duc­tion in pain com­pared to place­bo, and a 21% drop com­pared to bupi­va­caine. Pa­tient on HTX-011 took 38% few­er opi­oids post-surgery than the place­bo group, and 25% less than the bupi­va­caine group.

In a note to in­vestors last month, an­a­lysts at Jef­feries sug­gest­ed the piv­otal read­out for HTX-011 would be a “sig­nif­i­cant cat­a­lyst” for Heron. The com­pa­ny plans to file an NDA for the pro­gram lat­er this year.

The good news for the com­pa­ny fol­lows a reg­u­la­to­ry win just months ago, when Heron scored FDA ap­proval of Cin­van­ti to pre­vent nau­sea and vom­it­ing for pa­tients on chemo. It was the sec­ond an­ti-nau­sea drug in Heron’s prod­uct port­fo­lio, with Sus­tol ap­proved last Au­gust for the same in­di­ca­tion.

Shares at Heron have steadi­ly climbed 52% over the past 12 months. As of press time, the stock had climbed to $27.00 per share, up from yes­ter­day’s close of $21.50.

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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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.

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).