→ Bris­tol-My­ers Squibb has looped in a new part­ner to help scout new com­pa­ny cre­ation op­por­tu­ni­ties. As a lim­it­ed part­ner of Bio­Mo­tiv, the phar­ma gi­ant will work with the drug de­vel­op­ment ac­cel­er­a­tor to fund trans­la­tion­al ven­tures “in dis­ease ar­eas where un­met med­ical needs re­main” — and get first dibs to buy the re­sult­ing star­tups up­on lead iden­ti­fi­ca­tion. The deal gives Bris­tol-My­ers ac­cess to Bio­Mo­tiv’s net­work of aca­d­e­m­ic in­sti­tu­tions and their cut­ting-edge, ear­ly-stage dis­cov­er­ies, said Bruce Car, in­ter­im head of dis­cov­ery re­search.

One of Bio­Mo­tiv’s port­fo­lio com­pa­nies, Dual Ther­a­peu­tics, pre­vi­ous­ly en­ticed Bris­tol-My­ers Squibb to an on­col­o­gy pact that came with $255 mil­lion in mile­stones. The tie-up ap­peared to have fiz­zled, but Bris­tol-My­ers has con­tin­ued to get on the ground floor of biotech when­ev­er it could, for ex­am­ple by spon­sor­ing Bi­o­Lab‘s start­up fa­cil­i­ties.

→ It’s an­oth­er deal for Ger­man phar­ma Boehringer In­gel­heim. Af­ter ink­ing a re­search pact with MD An­der­son ear­li­er last month to cre­ate a “Vir­tu­al Re­search and De­vel­op­ment Cen­ter,” the com­pa­ny and Lupin Lim­it­ed have en­tered in­to a part­ner­ship for Lupin’s MEK in­hibitor com­pound (LNP3794) to be used in com­bi­na­tion with Boehringer’s KRAS in­hibitor for pa­tients with gas­troin­testi­nal and lung can­cers har­bor­ing a broad range of onco­genic KRAS mu­ta­tions.

The com­pa­ny says that “Lupin will re­ceive an up­front pay­ment of $20 mil­lion and po­ten­tial ad­di­tion­al pay­ments for suc­cess­ful achieve­ment of de­fined clin­i­cal, reg­u­la­to­ry and com­mer­cial mile­stones for a to­tal deal val­ue of more than $700 mil­lion.  Ad­di­tion­al­ly, Lupin will be en­ti­tled to re­ceive dou­ble-dig­it roy­al­ties on the sales of the prod­uct.”

→ Af­ter Ab­b­Vie read the last rites to their fi­nal, failed Ro­va-T clin­i­cal tri­al last week, the com­pa­ny is now col­lab­o­rat­ing with Idera Phar­ma to test com­bi­na­tions of an OX40 ag­o­nist (AB­BV-368) and a TLR-9 ag­o­nist (tilso­tolimod) — hop­ing to stim­u­late the im­mune sys­tem re­sult­ing in an­ti-tu­mor re­spons­es — in a Phase Ib study in pa­tients with re­cur­rent or metasta­t­ic head and neck squa­mous cell car­ci­no­ma (HN­SCC)

The study will test three sep­a­rate treat­ment arms: AB­BV-368 plus tilso­tolimod; AB­BV-368 plus tilso­tolimod and chemother­a­py (nab-pa­cli­tax­el); and AB­BV-368 plus tilso­tolimod, nab-pa­cli­tax­el and PD-1 an­tag­o­nist (AB­BV-181).

→ Los An­ge­les-based Puma Ther­a­peu­tics‘ drug Ner­l­ynx has been grant­ed or­phan drug sta­tus by the FDA for the treat­ment of breast can­cer pa­tients with brain metas­tases.

Back in May, in­vestor wor­ries cratered the com­pa­ny’s stock $PBYI when Q1 sales of the drug fell short of Wall Street es­ti­mates — with pa­tient dis­con­tin­u­a­tions run­ning high due to a poor tol­er­a­bil­i­ty pro­file, pre­dom­i­nate­ly re­lat­ing to di­ar­rhea. Ner­l­ynx was in-li­censed by Puma from Pfiz­er and was ap­proved by the FDA in Ju­ly 2017.

→ NICE has backed the NHS use of No­var­tis‘ gene ther­a­py Lux­tur­na for a rare, in­her­it­ed form of blind­ness in Eng­land and Wales. The one-off treat­ment, which was ap­proved in Eu­rope last No­vem­ber, has been sup­port­ed for use in chil­dren and adults who are liv­ing with vi­sion loss due to ge­net­ic mu­ta­tions in both copies of the RPE65 gene. No­var­tis bought the ex-US rights to the drug in 2018 for $170 mil­lion from Spark Ther­a­peu­tics.

→ The FDA has grant­ed re­gen­er­a­tive med­i­cine ad­vance ther­a­py (RMAT) des­ig­na­tion for Cam­bridge, Mass­a­chu­setts-based Ma­gen­ta Ther­a­peu­tics‘ MG­TA-456, a one-time cell ther­a­py for the treat­ment of mul­ti­ple in­her­it­ed meta­bol­ic dis­or­ders.

“The com­pa­ny is cur­rent­ly study­ing MG­TA-456 in a Phase II clin­i­cal tri­al in pa­tients old­er than 6 months of age with Hurler syn­drome, cere­bral adrenoleukody­s­tro­phy (cALD), metachro­mat­ic leukody­s­tro­phy (MLD) or globoid cell leukody­s­tro­phy (GLD) at four cen­ters of ex­cel­lence: Cincin­nati Chil­dren’s Hos­pi­tal, Duke Uni­ver­si­ty, Emory Uni­ver­si­ty and the Uni­ver­si­ty of Min­neso­ta.”

→ Re­genxbio has inked an op­tion and li­cense agree­ment for ex­clu­sive world­wide rights for Clear­side Bio­med­ical‘s SCS Mi­croin­jec­tor “for the de­liv­ery of RGX-314 to the supra­choroidal space to treat wet age-re­lat­ed mac­u­lar de­gen­er­a­tion (wet AMD), di­a­bet­ic retinopa­thy (DR) and oth­er con­di­tions for which an­ti-vas­cu­lar en­dothe­lial growth fac­tor (an­ti-VEGF) treat­ment is cur­rent­ly the stan­dard of care.”

The deal comes a lit­tle af­ter Clear­side Bio­med­ical an­nounced its pre­dic­tion that the FDA would re­ject its uveitis drug af­ter a re­quest for more da­ta.

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Former Sanofi chief Olivier Brandicourt, who departed his post with an unexpected early retirement last year, has made his move — as most C-suite executives inevitably do — to become a director on the board of a biopharma company.

RNAi player Alnylam is Brandicourt’s destination. Meanwhile, the Cambridge, Massachusetts-based drugmaker — which pioneered the first approval in the field — also disclosed the retirement of Alnylam co-founder Dr. Paul Schimmel from its board.

A coronavirus fear-induced bloodbath on the Nasdaq has not stopped Passage Bio from making a public debut — and an exuberant one.

By pricing an upsized offering at $18, the top of the range, the gene therapy biotech bagged $216 million from its IPO, 72% more than it’s originally penciled in.

The proceeds likely reflected confidence in Jim Wilson, who gathered all the tools he’s built over decades of gene therapy research to assemble the startup and teamed up with Frazier and OrbiMed to hone its focus on rare, monogenic disorders of the central nervous system. Just before the IPO, Deerfield partner Bruce Goldsmith took over from OrbiMed’s Stephen Squinto as CEO.